MA52665A - PROCESS FOR IMPROVING THE EXPRESSION OF FETAL HEMOGLOBIN - Google Patents

PROCESS FOR IMPROVING THE EXPRESSION OF FETAL HEMOGLOBIN

Info

Publication number
MA52665A
MA52665A MA052665A MA52665A MA52665A MA 52665 A MA52665 A MA 52665A MA 052665 A MA052665 A MA 052665A MA 52665 A MA52665 A MA 52665A MA 52665 A MA52665 A MA 52665A
Authority
MA
Morocco
Prior art keywords
expression
improving
fetal hemoglobin
hemoglobin
fetal
Prior art date
Application number
MA052665A
Other languages
French (fr)
Inventor
Riguo Fang
Fucai Liang
Jia Wang
Penghui Xia
Lingling Yu
Pengfei Yuan
Original Assignee
Edigene Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Edigene Inc filed Critical Edigene Inc
Publication of MA52665A publication Critical patent/MA52665A/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/18—Erythrocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0641—Erythrocytes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/10—Growth factors
    • C12N2501/125—Stem cell factor [SCF], c-kit ligand [KL]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/10—Growth factors
    • C12N2501/14—Erythropoietin [EPO]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/20—Cytokines; Chemokines
    • C12N2501/23—Interleukins [IL]
    • C12N2501/2303—Interleukin-3 (IL-3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/30—Hormones
    • C12N2501/38—Hormones with nuclear receptors
    • C12N2501/39—Steroid hormones
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/30—Hormones
    • C12N2501/38—Hormones with nuclear receptors
    • C12N2501/39—Steroid hormones
    • C12N2501/392—Sexual steroids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/999—Small molecules not provided for elsewhere
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Hematology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MA052665A 2017-10-27 2018-10-26 PROCESS FOR IMPROVING THE EXPRESSION OF FETAL HEMOGLOBIN MA52665A (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CN201711027708 2017-10-27

Publications (1)

Publication Number Publication Date
MA52665A true MA52665A (en) 2020-09-02

Family

ID=66246216

Family Applications (2)

Application Number Title Priority Date Filing Date
MA050844A MA50844A (en) 2017-10-27 2018-10-26 METHOD FOR INCREASING THE LEVEL OF FETAL HEMOGLOBIN EXPRESSION
MA052665A MA52665A (en) 2017-10-27 2018-10-26 PROCESS FOR IMPROVING THE EXPRESSION OF FETAL HEMOGLOBIN

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MA050844A MA50844A (en) 2017-10-27 2018-10-26 METHOD FOR INCREASING THE LEVEL OF FETAL HEMOGLOBIN EXPRESSION

Country Status (9)

Country Link
US (2) US20200384032A1 (en)
EP (2) EP3702459B1 (en)
JP (4) JP2021502062A (en)
CN (10) CN109722414A (en)
MA (2) MA50844A (en)
PH (2) PH12020550488A1 (en)
TW (2) TW201922777A (en)
WO (2) WO2019080917A1 (en)
ZA (2) ZA202002203B (en)

Families Citing this family (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2018007987A (en) * 2015-12-28 2019-01-10 Novartis Ag COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATIAS.
CN109722414A (en) * 2017-10-27 2019-05-07 博雅辑因（北京）生物科技有限公司 It is a kind of efficiently to prepare the method for mature erythrocyte and be used to prepare the culture medium of mature erythrocyte
CN109486814A (en) * 2017-10-31 2019-03-19 广东赤萌医疗科技有限公司 A kind of gRNA for repairing HBB1 point mutation, gene editing system, expression vector and gene editing kit
US12522811B2 (en) 2018-05-01 2026-01-13 The Children's Medical Center Corporation Enhanced BCL11A RNP / CRISPR delivery and editing using a 3XNLS-CAS9
CN112011576A (en) * 2019-05-31 2020-12-01 华东师范大学 Application of CRISPR gene editing technology in treating thalassemia
CN112391410B (en) * 2020-02-17 2024-04-02 华东师范大学 sgRNA and application thereof in repairing abnormal splicing of introns
CN110511909B (en) * 2019-07-29 2022-01-04 吉林大学 Growth factor composition for in vitro expansion of hematopoietic stem cells and application thereof
CN114364799B (en) * 2019-08-28 2024-07-19 甘李药业股份有限公司 Methods for editing the BCL11A gene in hematopoietic stem/progenitor cells
CN114222816A (en) * 2019-08-29 2022-03-22 广州辑因医疗科技有限公司 Method for efficiently repairing gene mutation of cricoid sideroblasts
CN112442516A (en) * 2019-08-29 2021-03-05 广州辑因医疗科技有限公司 Method for efficiently repairing gene mutation of cricoid sideroblasts
AU2020342207A1 (en) * 2019-09-04 2022-04-21 Edigene (Guangzhou) Inc. Method for evaluating gene editing therapy based on off-target assessment
JP7713233B2 (en) * 2019-09-11 2025-07-25 国立大学法人 東京大学 Albumin-free serum-free medium suitable for culturing human hematopoietic stem cells and albumin-free culture method
US20220380757A1 (en) * 2019-09-30 2022-12-01 The Children's Medical Center Corporation Bcl11a guide and base editor delivery
CN112746072A (en) * 2019-10-31 2021-05-04 广州瑞风生物科技有限公司 sgRNA for beta-hemoglobinopathy gene editing and application
CN111876416B (en) * 2020-07-01 2021-09-03 广州瑞风生物科技有限公司 Methods and compositions for activating gamma-globin gene expression
CN114149990A (en) * 2020-09-08 2022-03-08 甘李药业股份有限公司 Method for editing BCL11A gene in hematopoietic stem/progenitor cells
EP4255502A4 (en) * 2020-12-03 2025-07-16 Scribe Therapeutics Inc COMPOSITIONS AND METHODS FOR TARGETING BCL11A
US11970713B2 (en) * 2020-12-04 2024-04-30 Ocgene Therapeutics Corporation Method for long-term ex vivo maintenance or expansion of human erythroblast, human megakaryocyte-erythroid progenitor, or human common myeloid progenitor cell and application thereof
WO2022143694A1 (en) * 2020-12-28 2022-07-07 华东师范大学 Method for performing gene editing on single or multiple genes in cell, and product and use
CN113046330B (en) * 2021-03-23 2023-03-21 中吉智药(南京)生物技术有限公司 Lentivirus and medicine carrying erythroid gene editing system
EP4314308A4 (en) * 2021-04-02 2026-05-20 Univ Shanghai Technology Gene therapy for the treatment of beta-hemoglobinopathia
CN114032240A (en) * 2021-11-02 2022-02-11 珠海横琴爱姆斯坦生物科技有限公司 A method for improving gene knockout efficiency
CN116179547A (en) * 2021-11-26 2023-05-30 博雅缉因（北京）生物科技有限公司 Method for improving WAS protein expression in human cells
WO2023107675A2 (en) * 2021-12-10 2023-06-15 The Children's Medical Center Corporation Combination bcl11a enhancer editing
CN114317436A (en) * 2021-12-30 2022-04-12 中国人民解放军军事科学院军事医学研究院 Method for reactivating human gamma-globin gene expression
CN115838688B (en) * 2022-01-24 2025-10-10 国家卫生健康委科学技术研究所 A culture medium for expanding hematopoietic stem cells and its application
CN115960968A (en) * 2022-09-30 2023-04-14 珠海横琴爱姆斯坦生物科技有限公司 Method for preparing genetic engineering pluripotent stem cells of BCL11A erythroid enhancer homozygous mutation
CN116042520A (en) * 2022-11-08 2023-05-02 唐颐控股(深圳)有限公司 A kind of in vitro expansion culture system and method of hematopoietic stem cells
CN116355960A (en) * 2023-02-17 2023-06-30 中国科学院动物研究所 Construction and application of a mouse model that accurately differentiates functional hematopoietic stem cells
CN116445402B (en) * 2023-05-09 2025-04-25 中国科学院生态环境研究中心 Erythroid progenitor cell expansion culture medium, expansion culture method and application
CN116426472A (en) * 2023-06-08 2023-07-14 呈诺再生医学科技(北京)有限公司 Induced differentiation system for promoting hematopoietic stem cells or hematopoietic progenitor cells to differentiate into erythrocytes and application thereof
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2026104502A1 (en) * 2024-11-13 2026-05-21 Institut National de la Santé et de la Recherche Médicale Epigenetic reactivation of gamma-globin expression as a novel curative option for β-hemoglobinopathies
CN121422063A (en) * 2025-12-31 2026-01-30 广州瑞风生物科技股份有限公司 A pharmaceutical composition for treating thalassemia and its application

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN100506978C (en) * 2001-09-21 2009-07-01 中国人民解放军军事医学科学院野战输血研究所 Hemapoietic stem/ancestral cell enriching method and its in vitro directional induction and differentiation
CN101045914A (en) * 2006-03-29 2007-10-03 中国人民解放军军事医学科学院野战输血研究所 Extracorporeal induction process for differentiating hemopoietic stem/ancestral cell into mature red blood cell and its application
WO2011072086A1 (en) * 2009-12-08 2011-06-16 Hemaquest Pharmaceuticals, Inc. Methods and low dose regimens for treating red blood cell disorders
EP2836226B1 (en) * 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
CN102643784B (en) * 2012-03-29 2016-05-25 中国人民解放军第四军医大学 A kind of amplification in vitro system of hematopoietic stem/progenitor
ES2728782T3 (en) 2012-05-25 2019-10-28 Univ California Methods and compositions for RNA-directed target DNA modification and for RNA-directed transcription modulation
CN116622704A (en) 2012-07-25 2023-08-22 布罗德研究所有限公司 Inducible DNA-binding proteins and genome interference tools and their applications
CN103667188B (en) * 2012-09-21 2016-02-24 北京市红十字血液中心 One prepares ripe erythrocytic method
EP4397759A3 (en) 2012-10-23 2024-10-09 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
ES2708948T3 (en) * 2012-11-27 2019-04-12 Childrens Medical Ct Corp Distal regulatory elements of bcl11a as targets for the reinduction of fetal hemoglobin
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
ES2721936T3 (en) * 2013-11-13 2019-08-06 Childrens Medical Center Regulation of nuclease-mediated gene expression
US10072067B2 (en) * 2014-01-30 2018-09-11 Children's Hospital Medical Center Fetal hemoglobin for genetic correction of sickle cell disease
CN106794260B (en) * 2014-04-25 2021-02-23 儿童医疗中心有限公司 Compositions and methods for treating hemoglobinopathies
CN113699113A (en) * 2014-09-16 2021-11-26 桑格摩治疗股份有限公司 Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells
EP3288570A4 (en) * 2015-04-29 2018-11-21 Fred Hutchinson Cancer Research Center Modified stem cells and uses thereof
EP3763814A1 (en) * 2015-05-08 2021-01-13 The Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
CN104877965B (en) * 2015-05-27 2018-04-20 上海厚东生物科技有限公司 A kind of method for preparing mature erythrocyte
US9957501B2 (en) * 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
CN105238758A (en) * 2015-09-17 2016-01-13 中国科学院广州生物医药与健康研究院 In-vitro hematopoietic stem cell/ progenitor cell acquisition method
MX2018007987A (en) * 2015-12-28 2019-01-10 Novartis Ag COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATIAS.
CN105854017A (en) * 2016-03-01 2016-08-17 扬州大学 Reagent for treating beta-thalassemia and application thereof
CN105754939A (en) * 2016-04-15 2016-07-13 广州市天河诺亚生物工程有限公司 Method for improving directional differentiation effects on erythroid progenitor cells in umbilical cord blood
JP6974349B2 (en) * 2016-04-18 2021-12-01 クリスパー セラピューティクス アクチェンゲゼルシャフト Materials and methods for the treatment of hemoglobin abnormalities
EP3596217A1 (en) * 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CN107151655A (en) * 2017-04-10 2017-09-12 中国人民解放军第三O七医院 A kind of method of cell amplification
CN109722414A (en) * 2017-10-27 2019-05-07 博雅辑因（北京）生物科技有限公司 It is a kind of efficiently to prepare the method for mature erythrocyte and be used to prepare the culture medium of mature erythrocyte

Also Published As

Publication number Publication date
ZA202002203B (en) 2023-05-31
EP3702459A4 (en) 2021-07-21
CN111278976A (en) 2020-06-12
CN111278977B (en) 2022-03-22
CN109722415A (en) 2019-05-07
CN109722414A (en) 2019-05-07
JP2022180511A (en) 2022-12-06
EP3702458A1 (en) 2020-09-02
TW201930592A (en) 2019-08-01
EP3702459B1 (en) 2023-10-04
JP2021500058A (en) 2021-01-07
US20200384032A1 (en) 2020-12-10
JP2021502062A (en) 2021-01-28
CN109735497A (en) 2019-05-10
CN109722415B (en) 2021-01-26
JP2022180510A (en) 2022-12-06
EP3702459A1 (en) 2020-09-02
PH12020550487A1 (en) 2021-03-22
TW201922777A (en) 2019-06-16
ZA202002204B (en) 2022-10-26
US20200339979A1 (en) 2020-10-29
WO2019080920A1 (en) 2019-05-02
WO2019080917A1 (en) 2019-05-02
EP3702458A4 (en) 2021-08-25
CN111278977A (en) 2020-06-12
CN109735497B (en) 2021-11-02
CN115747165A (en) 2023-03-07
CN114921415A (en) 2022-08-19
MA50844A (en) 2020-09-02
CN114875026A (en) 2022-08-09
PH12020550488A1 (en) 2021-03-22
CN109735574A (en) 2019-05-10
CN111278976B (en) 2022-03-01
CN114657140A (en) 2022-06-24

Similar Documents

Publication Publication Date Title
EP3702459A4 (en) PROCESS FOR IMPROVING THE EXPRESSION OF FETAL HEMOGLOBIN
MA50169A (en) PROCESS FOR THE PRODUCTION OF AN ELECTROCATALYZER
EP3353841A4 (en) PROCESS FOR THE PREPARATION AND APPLICATION OF CARBON-SELENIUM COMPOSITES
EP3302794A4 (en) EPOXIDATION PROCESS
MA52906A (en) AMG 416 PREPARATION PROCESS
EP3443094A4 (en) METHODS OF REDUCING EXPRESSION OF C9ORF72
EP3383436A4 (en) IMPROVED PROCESS FOR THE PREPARATION OF INDOCYANINE GREEN
EP3695433A4 (en) CROSS-SECTION SAMPLE PREPARATION PROCESS
IL260730B (en) Process for the preparation of terpinan-4-ol
EP3398957A4 (en) PROCESS FOR SYNTHESIZING ETTEAL ALLOYDE
EP3364990A4 (en) PROCESS FOR INCREASING THE BIOACTIVITY OF ASHWAGANDA EXTRACTS
EP3395530A4 (en) PROCESS FOR THE PRODUCTION OF STRUCTURE
EP3766869A4 (en) SIMPLE PROCESS FOR PREPARING AVIBACTAM
EP3630738A4 (en) OZANIMOD PRODUCTION PROCESS
EP3356033A4 (en) PROCESS FOR THE CATALYTIC HYDROGENATION OF HALONITROAROMATIC
EP3341359A4 (en) PROCESS FOR PREPARING APREMILAST
MA42423A (en) COATING PREPARATION PROCESS
EP3768487A4 (en) PROCESS FOR MICROMOLDINGS OF ARTICLES
EP3720845A4 (en) ELAGOLIX PRODUCTION PROCESS
EP3497088A4 (en) EPOXIDATION PROCESS
EP3423402A4 (en) PROCESS FOR THE SYNTHESIS OF NANODIAMANTS
EP3792296C0 (en) PROCESS FOR THE SYNTHESIS OF POLYETHERS
EP3447042A4 (en) PROCESS FOR THE PRODUCTION OF 1,2-DICHLOROHEXAFLUOROCYCLOPENTENE
EP3718992A4 (en) OLEFIN OLIGOMERIZATION PROCESS
EP3466913A4 (en) PROCESS FOR OBTAINING 1-OCTANOL